Content » Vol 84, Issue 5

Clinical Report

Enzyme Replacement Therapy in Severe Fabry Disease with Renal Failure: A 1-year Follow-up

Dionysios Tsambaos, Elisabeth Chroni, Antonis S. Manolis, Alexandra Monastirli, Efi Pasmatzi, Theophilos Sakkis, Periklis Davlouros, Dimitrios Goumenos, Aggeliki Katrivanou, Sophia Georgiou
DOI: 10.1080/00015550410030682


Wepresent here the course of clinical response of a 53-year-oldhaemodialysed Fabry patient who received recombinant human α-galactosidase A at a dose of 1 mg/kg every other week over a period of 1 year. The therapy was well tolerated by the patient, who revealedan impressive favourable cutaneous, gastrointestinal, neurological andpsychiatric response and a dramatic improvement in his quality of life,but no improvement in cardiac and renal function.


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